Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells

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Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells

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Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

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Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases

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Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

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Chemogenetics as a neuromodulatory approach to treating neuropsychiatric diseases and disorders

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Beyond cancer cells: Targeting the tumor microenvironment with gene therapy and armed oncolytic virus

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Dual-AAV delivering split prime editor system for in vivo genome editing

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Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor

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Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo

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CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genes

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Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction

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Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

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Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency

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Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

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Bi-directional gene activation and repression promote ASC differentiation and enhance bone healing in osteoporotic rats

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Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells

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Interplay between Triadin and Calsequestrin in the Pathogenesis of CPVT in the Mouse

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Gene-Loaded Nanoparticle-Coated Sutures Provide Effective Gene Delivery to Enhance Tendon Healing

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IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification

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